Huntington’s
Nine months of an investigative treatment appears to slow Huntington's progression in early-stage patients, interim Phase 1 data from an Australian substudy suggests. Huntington's is a progressive disorder caused by a genetic change that produces a toxic form of huntingtin, a protein that harms nerve cells. New interim results indicate the oral candidate SKY-0515 slowed [...]
Northern Scotland has one of the world's highest Huntington's disease rates, more than five times the estimated global average, new research shows. For the first time in 35 years, scientists have accurately quantified the number of people in the region carrying the gene that causes the condition. Using NHS family-based records, researchers identified more than [...]
A man who lost three family members to Huntington's disease is completing a 100km challenge to raise awareness and break stigma around the condition. Mark Barrett, 38, from Howwood, Scotland, is taking on four half marathons and a triathlon over nine weeks after losing his father Frank, grandfather John and uncle Raymond to the inherited [...]
Gene therapy slowed Huntington's disease progression by 75 per cent in a recent trial, in what researchers describe as the first effective treatment for the condition. The therapy means that decline expected in one year would instead take four years, potentially giving patients decades of better quality life, researchers said. Researchers from University [...]
Children of people with Huntington's disease are more likely to experience trauma growing up, which may contribute to mental health problems in later life. A study found that emotional abuse in childhood was the strongest predictor of psychological distress in adulthood among those from Huntington’s-affected families. Huntington’s disease is a progressive genetic brain disorder that [...]
A man living in Worcestershire has fulfilled one of his lifelong ambitions to spend an afternoon driving around the Malvern Hills in a beautiful classic sports car, despite living with a complex neurological condition. Mark, 59, who is a resident at the Inspire Neurocare service in Worcester, asked if it would be possible to make [...]
Transplanting healthy human brain support cells into adult mice with Huntington’s disease slowed motor and cognitive decline and extended lifespan, researchers have found. The study suggests an alternative approach to treating Huntington’s – a hereditary condition that damages nerve cells – by replacing diseased glial cells rather than focusing solely on preserving neurons. Researchers observed [...]
Smartphone assessments could help researchers more precisely detect progression in Huntington’s disease, potentially improving the efficiency of clinical trials, new research suggests. A five-minute test delivered via a custom app calculates a Huntington’s Disease Digital Motor Score (HDDMS) using five simple assessments of movement control – including balance, finger tapping and involuntary movements. Huntington’s disease [...]
Gene therapy company uniQure recently announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to AMT-130 for the treatment of Huntington’s disease. There are currently no disease-modifying therapies available for Huntington's Disease, highlighting the urgent need for innovation. uniQure has stated that AMT-130's Breakthrough Therapy designation has been granted based on clinical [...]
Results from the Phase 2 PIVOT-HD study of PTC518 (votoplam) in Stage 2 and Stage 3 Huntington's disease (HD) patients demonstrated that the treatment met its primary endpoint of reduction in blood Huntingtin (HTT) protein levels. The treatment showed a reduction in HTT protein levels at Week 12 and favourable safety and tolerability. The 12-month [...]
